Your Questions, Answered: 2026 Q&A
About this episode
In this episode, Stan Crooke answers questions from the nano-rare community regarding drug safety, ASO administration, and commercialization plans.
Your questions, answered. n-Lorem CEO, Stan Crooke, answers questions directly from the nano-rare community in this special Q&A episode. How often is Drug safety monitored? When is an existing ASO made available to others? Not capping accepted applications and more. Do you have additional questions? Email podcast@nlorem.org. On This Episode We Discuss: 5:40 - What is the frequency range of intrathecal and/or LP administrations? Do the treatments not typically cross the blood brain barrier? 7:06 - How do you administer the ASO to animals - is it according to how you plan to administer to the patient? 7:36 - How do you evaluate the potential on-target toxicity if the nonclinical species does not contain the targeted sequence? 9:35 - How often is safety monitored? Is it only monitored quarterly at the Data/Drug Safety Monitoring Board (DSMB) meetings? 11:03 - How long after an ASO is developed for one child, is it offered to other children? Of course, we know that they will have to submit an application. Does the ASO become potentially available for other children after the first child's 2-year trial is over? Or is it potentially available after just a few doses to confirm…
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- Realities of the Nano-rare: Episode 1 with Sarah Glass
- Miracles of Science #2: Induced Pluripotent Stem Cells
- Where Emerging FDA Draft Guidance may Lead for Nano-rare Patients
- Reflections from the n-Lorem Chairman | 2026
- Miracles of Science: Genomics
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